HCPW P feedback from CHMP Presented by: Ftima Ventura (CHMP) 18 - - PowerPoint PPT Presentation

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HCPW P feedback from CHMP

Presented by: Fátima Ventura (CHMP) 18 April 2018

HCPWP feedback from CHMP – April 2018 1

Sum m ary

• CHMP opinions (overview Oct 2017 – Mar 2018)

– New medicines – Scientific Advices/ Protocol Assistance – PRIME eligibility

• HCP/ P input provided in the context of CHMP activities

Nam e DS I ndication Adynovi rurioctocog alfa pegol Haemophilia A Ad hoc expert group ( included 2 Patients’ representatives)

Post-authorisation safety study (PASS): In order to investigate potential effects - Q1 2019

Hem libra emicizumab Haemophilia A - factor VIII inhibitors

EP/ EM - Physician, Patient/ Carer, and Laboratory professionals educational material

I ntrarosa prasterone Vulvar and vaginal atrophy in postmenopausal women Crysvita burosumab X-linked hypophosphataemia Lam zede velmanase alfa non-neurological manifestations of mild to moderate alpha- mannosidosis Ad hoc expert group ( included 2 Patients’ representatives)

Post-MA: Long-term data on effectiveness and safety data from a registry of patients + Annual reports/ annual re-assessment + Paediatric Study - Final Study report: November 2020

2

Positive opinion on new m edicines – Oct 1 7 – Mar 1 8 EC EC EC EC EC

HCPWP feedback from CHMP – April 2018

Nam e DS I ndication Fasenra benralizumab Severe eosinophilic asthma Jorveza budesonide Eosinophilic esophagitis Alofisel darvadstrocel Complex perianal fistulas in patients with Crohn’s disease

EP/ EM - Guide for pharmacists with instructions on the appropriate reception and storage. Guide (video) for surgeons and other HCP involved in the preparation and administration. Guide for surgeons and other HCP describing the method of administration and providing information on potential for microbial information and advice on steps to follow in case a positive culture is identified. Relevant information on the risk of medication errors and the potential for transmission of infectious agents and details on how to minimise these, including reception, storage and administration instructions (i.e. fistula conditioning, preparation and injection). Post-MA: To follow-up on the efficacy of Alofisel, the MAH should submit the results of a Phase III randomised double-blind, placebo-controlled study investigating a single administration - 2Q/ 3Q 2022

Ocrevus

• crelizumab

Relapsing forms of MS and primary progressive MS SAG ( included 2 Patients’ representatives)

3

Positive opinion on new m edicines – Oct 1 7 – Mar 1 8 EC EC EC EC

HCPWP feedback from CHMP – April 2018

Nam e DS I ndication Ozem pic semaglutide Type 2 diabetes Steglatro ertugliflozin Type 2 diabetes Seglurom et ertugliflozin / metformin Type 2 diabetes Steglujan ertugliflozin / sitagliptin Type 2 diabetes Am glidia glibenclamide Neonatal diabetes

Measure to minimise medication errors - Number of presentations for the two different dose strengths and syringes EM - Visual educational material for physicians and pharmacists

Alkindi hydrocortisone Replacement therapy of adrenal insufficiency in infants, children and adolescents

Positive opinion on new m edicines – Oct 1 7 – Mar 1 8 EC EC EC EC EC EC

HCPWP feedback from CHMP – April 2018 4

Nam e DS I ndication Prevym is letermovir Prophylaxis of CMV reactivation and disease in adult CMV- seropositive recipients of HSCT

Post-MA: In order to optimise the sterility assurance level implement the measures concerning development, validation and introduction of terminal sterilisation.

Shingrix herpes zoster vaccine (recombinant, adjuvanted) Prevention of herpes zoster (HZ) and post-herpetic neuralgia (PHN), in adults > 50 years Alpivab peramivir Uncomplicated influenza Juluca dolutegravir / rilpivirine HIV infection Mylotarg gemtuzumab

• zogamicin

Acute myeloid leukaemia Rubraca rucaparib Relapsed or progressive

• varian cancer

SAG ( included 2 Patients’ representatives)

Positive opinion on new m edicines – Oct 1 7 – Mar 1 8 EC EC EC EC EC EC

5 HCPWP feedback from CHMP – April 2018

HCPWP feedback from CHMP – April 2018 6

Scientific Advice/ Protocol assis. ( Oct 1 7 – Mar 1 8 )

117 84 20 7 14 50 100 150

Chemicals Biologicals ATMP Innovative HTA

Number SA

Drug substance type

78 9 2 25 13 26 22 59 20 40 60 80 100 Number SA

Therapeutic area

HCPWP feedback from CHMP – April 2018 7

PRI ME eligibility – Oct 1 7 – Mar 2 0 1 8

Name Substance type Therapeutic área Therapeutic indication Data of eligibility granted Entrectinib Chemical Oncology Treatment of NTRK fusion- positive, locally advanced or metastatic solid tumours in adult and paediatric patients who have either progressed following prior therapies or who have no acceptable standard therapy 10-2017 Humanized antibody targeting B cell maturation antigen conjugated with maleimidocaproyl monomethyl auristatin F Biological Oncology Treatment of relapsed and refractory multiple myeloma patients whose prior therapy included a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 antibody 10-2017 Human immunoglobulin G1 constant region - human ectodysplasin- A1 receptor-binding domain fusion protein Biological Dermatology Treatment of X-linked hypohidrotic ectodermal dysplasia 10-2017

HCPWP feedback from CHMP – April 2018 8

PRI ME eligibility – Oct 1 7 – Mar 2 0 1 8

Name Substance type Therapeutic área Therapeutic indication Data of eligibility granted Autologous T lymphocyte-enriched population of cells transduced with a lentiviral vector encoding a chimeric antigen receptor targeting human B cell maturation antigen with 4-1BB and CD3- zeta intracellular signalling domains (bb2121) Advanced therapy Oncology Treatment of relapsed and refractory multiple myeloma patients whose prior therapy included a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 antibody 11-2017 LR12 Chemical Infectious Diseases Treatment of Septic Shock 11-2017 Recombinant humanised monoclonal IgG2 lambda antibody against human sclerostin (BPS804) Biological Other Treatment of osteogenesis imperfecta types I, III and IV 11-2017

HCPWP feedback from CHMP – April 2018 9

PRI ME eligibility – Oct 1 7 – Mar 2 0 1 8

Name Substance type Therapeutic área Therapeutic indication Data of eligibility granted Adenovirus associated viral vector serotype 8 containing the human CNGB3 gene (AAV2/8- hCARp.hCNGB3) Advanced therapy Oncology Treatment of achromatopsia associated with defects in CNGB3 02-2018 Lumasiran Chemical Uro-nephrology Treatment of Primary Hyperoxaluria Type 1 03-20178

HCPWP feedback from CHMP – April 2018 10

I nteraction betw een CHMP & HCP & Patients

Nam e I ndication Consultation Outcom e Adynovi haemophilia A Ad-Hoc (2 Pts) √ Ocrevus multiple sclerosis SAG (2 Pts) √ Alcover ( A-3 2 ) alcohol dependence Ad-Hoc X Fanaptum schizophrenia Ad-Hoc (2 Pts) X Lamzede alpha-mannosidosis Ad-Hoc (2 Pts) √ Nerlynx early breast cancer SAG (2 Pts) X Onzeald advanced breast cancer SAG (2 Pts) X Opdivo ( ext. ind.)

• met. colorectal cancer

SAG (2 Pts) W ithdraw n Rubraca

• prog. ovarian cancer

SAG (2 Pts) √ Raxone ( ext. ind.; after re-exm ) Duchenne muscular dystrophy SAG (2 Pts)/ CHMP X Eladynos

• steoporosis

Ad-Hoc (2 Pts) X Aplidin ( after re-exam ) multiple myeloma SAG (2 Pts) X